RU2019140862A - Варианты сульфамидазы (sgsh), векторы, композиции и способы, и применения для лечения мукополисахаридоз типа iiia (mps iiia) - Google Patents
Варианты сульфамидазы (sgsh), векторы, композиции и способы, и применения для лечения мукополисахаридоз типа iiia (mps iiia) Download PDFInfo
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- RU2019140862A RU2019140862A RU2019140862A RU2019140862A RU2019140862A RU 2019140862 A RU2019140862 A RU 2019140862A RU 2019140862 A RU2019140862 A RU 2019140862A RU 2019140862 A RU2019140862 A RU 2019140862A RU 2019140862 A RU2019140862 A RU 2019140862A
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- 239000000203 mixture Substances 0.000 title claims 27
- 102100027661 N-sulphoglucosamine sulphohydrolase Human genes 0.000 title 3
- 208000005340 mucopolysaccharidosis III Diseases 0.000 title 2
- 101000651201 Homo sapiens N-sulphoglucosamine sulphohydrolase Proteins 0.000 title 1
- 108010006140 N-sulfoglucosamine sulfohydrolase Proteins 0.000 title 1
- 208000025816 Sanfilippo syndrome type A Diseases 0.000 title 1
- 208000011045 mucopolysaccharidosis type 3 Diseases 0.000 title 1
- 208000012226 mucopolysaccharidosis type IIIA Diseases 0.000 title 1
- 239000013598 vector Substances 0.000 title 1
- KIUKXJAPPMFGSW-DNGZLQJQSA-N (2S,3S,4S,5R,6R)-6-[(2S,3R,4R,5S,6R)-3-Acetamido-2-[(2S,3S,4R,5R,6R)-6-[(2R,3R,4R,5S,6R)-3-acetamido-2,5-dihydroxy-6-(hydroxymethyl)oxan-4-yl]oxy-2-carboxy-4,5-dihydroxyoxan-3-yl]oxy-5-hydroxy-6-(hydroxymethyl)oxan-4-yl]oxy-3,4,5-trihydroxyoxane-2-carboxylic acid Chemical compound CC(=O)N[C@H]1[C@H](O)O[C@H](CO)[C@@H](O)[C@@H]1O[C@H]1[C@H](O)[C@@H](O)[C@H](O[C@H]2[C@@H]([C@@H](O[C@H]3[C@@H]([C@@H](O)[C@H](O)[C@H](O3)C(O)=O)O)[C@H](O)[C@@H](CO)O2)NC(C)=O)[C@@H](C(O)=O)O1 KIUKXJAPPMFGSW-DNGZLQJQSA-N 0.000 claims 6
- SQDAZGGFXASXDW-UHFFFAOYSA-N 5-bromo-2-(trifluoromethoxy)pyridine Chemical compound FC(F)(F)OC1=CC=C(Br)C=N1 SQDAZGGFXASXDW-UHFFFAOYSA-N 0.000 claims 6
- 229920001287 Chondroitin sulfate Polymers 0.000 claims 6
- NBIIXXVUZAFLBC-UHFFFAOYSA-L Phosphate ion(2-) Chemical compound OP([O-])([O-])=O NBIIXXVUZAFLBC-UHFFFAOYSA-L 0.000 claims 6
- 229940059329 chondroitin sulfate Drugs 0.000 claims 6
- 201000003146 cystitis Diseases 0.000 claims 6
- NBIIXXVUZAFLBC-UHFFFAOYSA-M dihydrogenphosphate Chemical compound OP(O)([O-])=O NBIIXXVUZAFLBC-UHFFFAOYSA-M 0.000 claims 6
- 229920002674 hyaluronan Polymers 0.000 claims 5
- 229960003160 hyaluronic acid Drugs 0.000 claims 5
- 208000027866 inflammatory disease Diseases 0.000 claims 5
- 239000008363 phosphate buffer Substances 0.000 claims 5
- 150000003839 salts Chemical class 0.000 claims 5
- 239000004480 active ingredient Substances 0.000 claims 4
- 230000001684 chronic effect Effects 0.000 claims 3
- 239000003792 electrolyte Substances 0.000 claims 3
- 208000005615 Interstitial Cystitis Diseases 0.000 claims 2
- 230000001580 bacterial effect Effects 0.000 claims 2
- 210000001635 urinary tract Anatomy 0.000 claims 2
- 206010063057 Cystitis noninfective Diseases 0.000 claims 1
- 201000005661 acute cystitis Diseases 0.000 claims 1
- 230000001154 acute effect Effects 0.000 claims 1
- 229910052783 alkali metal Inorganic materials 0.000 claims 1
- -1 alkali metal salt Chemical class 0.000 claims 1
- 239000007853 buffer solution Substances 0.000 claims 1
- 201000003139 chronic cystitis Diseases 0.000 claims 1
- 230000000069 prophylactic effect Effects 0.000 claims 1
- 201000007608 radiation cystitis Diseases 0.000 claims 1
- 230000000306 recurrent effect Effects 0.000 claims 1
- 230000001225 therapeutic effect Effects 0.000 claims 1
Classifications
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
- A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/0075—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the delivery route, e.g. oral, subcutaneous
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/0083—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the administration regime
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
- C07K2319/02—Fusion polypeptide containing a localisation/targetting motif containing a signal sequence
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y310/00—Hydrolases acting on sulfur-nitrogen bonds (3.10)
- C12Y310/01—Hydrolases acting on sulfur-nitrogen bonds (3.10) acting on sulfur-nitrogen bonds (3.10.1)
- C12Y310/01001—N-Sulfoglucosamine sulfohydrolase (3.10.1.1)
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biotechnology (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Medicinal Chemistry (AREA)
- Molecular Biology (AREA)
- Biomedical Technology (AREA)
- General Engineering & Computer Science (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Biochemistry (AREA)
- Epidemiology (AREA)
- Microbiology (AREA)
- Virology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Neurosurgery (AREA)
- Neurology (AREA)
- Immunology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Enzymes And Modification Thereof (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762505423P | 2017-05-12 | 2017-05-12 | |
| US62/505,423 | 2017-05-12 | ||
| PCT/US2018/032454 WO2018209317A1 (en) | 2017-05-12 | 2018-05-11 | Sulfamidase (sgsh) variants, vectors, compositions and methods and uses for treating mucopolysaccharidosis type iiia (mps iiia) |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| RU2019140862A true RU2019140862A (ru) | 2021-06-15 |
| RU2019140862A3 RU2019140862A3 (enExample) | 2021-08-17 |
Family
ID=64104969
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| RU2019140862A RU2019140862A (ru) | 2017-05-12 | 2018-05-11 | Варианты сульфамидазы (sgsh), векторы, композиции и способы, и применения для лечения мукополисахаридоз типа iiia (mps iiia) |
Country Status (10)
| Country | Link |
|---|---|
| US (1) | US12221635B2 (enExample) |
| EP (1) | EP3622060A4 (enExample) |
| JP (1) | JP7196099B2 (enExample) |
| CN (1) | CN110809626A (enExample) |
| AU (1) | AU2018265869C1 (enExample) |
| BR (1) | BR112019023869A2 (enExample) |
| CA (1) | CA3061925A1 (enExample) |
| MX (1) | MX2019013528A (enExample) |
| RU (1) | RU2019140862A (enExample) |
| WO (1) | WO2018209317A1 (enExample) |
Families Citing this family (9)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN108728495A (zh) * | 2018-05-31 | 2018-11-02 | 深圳市免疫基因治疗研究院 | 一种Sanfilippo A综合症慢病毒载体、慢病毒及其制备方法和应用 |
| ES3035673T3 (en) * | 2018-12-20 | 2025-09-08 | Esteve Pharmaceuticals Sa | Recombinant vectors for the long term treatment of mucchopolysacharidosis |
| WO2021011841A1 (en) * | 2019-07-18 | 2021-01-21 | Lysogene | Compositions and methods for the treatment of sanfilippo disease and other disorders |
| MX2022006188A (es) * | 2019-11-22 | 2022-08-22 | Childrens Hospital Philadelphia | Variantes del vector viral adenoasociado. |
| JP2023517230A (ja) * | 2020-03-11 | 2023-04-24 | リモーター セラピューティクス インコーポレイテッド | 中枢神経系全体にわたってタンパク質を散在させるための方法および材料 |
| CN111718947B (zh) * | 2020-06-18 | 2022-08-23 | 舒泰神（北京）生物制药股份有限公司 | 用于治疗ⅲa或ⅲb型粘多糖贮积症的腺相关病毒载体及用途 |
| WO2023086939A1 (en) * | 2021-11-12 | 2023-05-19 | Amicus Therapeutics, Inc. | Compositions and methods for treating mucopolysaccharidosis iiia |
| JP2023159049A (ja) * | 2022-04-19 | 2023-10-31 | Ｊｃｒファーマ株式会社 | マンノース－６－リン酸の数を減少させた糖蛋白質 |
| CN115029360B (zh) * | 2022-05-30 | 2024-08-02 | 上海勉亦生物科技有限公司 | 用于治疗粘多糖贮积症iiia型的转基因表达盒 |
Family Cites Families (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5863782A (en) * | 1995-04-19 | 1999-01-26 | Women's And Children's Hospital | Synthetic mammalian sulphamidase and genetic sequences encoding same |
| TWI688395B (zh) * | 2010-03-23 | 2020-03-21 | 英翠克頌公司 | 條件性表現治療性蛋白質之載體、包含該載體之宿主細胞及彼等之用途 |
| EP2394667A1 (en) | 2010-06-10 | 2011-12-14 | Laboratorios Del Dr. Esteve, S.A. | Vectors and sequences for the treatment of diseases |
| US9849195B2 (en) * | 2011-03-31 | 2017-12-26 | University Of Iowa Research Foundation | Methods and compositions for treating brain diseases |
| US20170191041A1 (en) * | 2014-07-11 | 2017-07-06 | Biostrategies LC | Materials and methods for treating disorders associated with sulfatase enzymes |
-
2018
- 2018-05-11 WO PCT/US2018/032454 patent/WO2018209317A1/en not_active Ceased
- 2018-05-11 MX MX2019013528A patent/MX2019013528A/es unknown
- 2018-05-11 US US16/611,458 patent/US12221635B2/en active Active
- 2018-05-11 AU AU2018265869A patent/AU2018265869C1/en active Active
- 2018-05-11 EP EP18798587.4A patent/EP3622060A4/en active Pending
- 2018-05-11 CN CN201880043043.4A patent/CN110809626A/zh active Pending
- 2018-05-11 RU RU2019140862A patent/RU2019140862A/ru unknown
- 2018-05-11 CA CA3061925A patent/CA3061925A1/en active Pending
- 2018-05-11 BR BR112019023869A patent/BR112019023869A2/pt unknown
- 2018-05-11 JP JP2019562293A patent/JP7196099B2/ja active Active
Also Published As
| Publication number | Publication date |
|---|---|
| CA3061925A1 (en) | 2018-11-15 |
| WO2018209317A1 (en) | 2018-11-15 |
| JP7196099B2 (ja) | 2022-12-26 |
| BR112019023869A2 (pt) | 2020-06-09 |
| JP2020519629A (ja) | 2020-07-02 |
| MX2019013528A (es) | 2020-07-14 |
| US12221635B2 (en) | 2025-02-11 |
| EP3622060A1 (en) | 2020-03-18 |
| AU2018265869C1 (en) | 2025-01-30 |
| CN110809626A (zh) | 2020-02-18 |
| AU2018265869B2 (en) | 2024-10-24 |
| RU2019140862A3 (enExample) | 2021-08-17 |
| US20210324354A1 (en) | 2021-10-21 |
| AU2018265869A1 (en) | 2019-11-21 |
| EP3622060A4 (en) | 2021-02-17 |
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